PT3283500T - Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão - Google Patents

Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão

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Publication number
PT3283500T
PT3283500T PT167772912T PT16777291T PT3283500T PT 3283500 T PT3283500 T PT 3283500T PT 167772912 T PT167772912 T PT 167772912T PT 16777291 T PT16777291 T PT 16777291T PT 3283500 T PT3283500 T PT 3283500T
Authority
PT
Portugal
Prior art keywords
compositions
methods
muscular dystrophy
exon skipping
girdle muscular
Prior art date
Application number
PT167772912T
Other languages
English (en)
Portuguese (pt)
Original Assignee
Univ Chicago
Univ Northwestern
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Univ Chicago, Univ Northwestern filed Critical Univ Chicago
Publication of PT3283500T publication Critical patent/PT3283500T/pt

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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C12N15/1138—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing against receptors or cell surface proteins
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09—Recombinant DNA-technology
    • C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/10—Type of nucleic acid
    • C12N2310/11—Antisense
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/315—Phosphorothioates
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/31—Chemical structure of the backbone
    • C12N2310/318—Chemical structure of the backbone where the PO2 is completely replaced, e.g. MMI or formacetal
    • C12N2310/3181—Peptide nucleic acid, PNA
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/321—2'-O-R Modification
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/32—Chemical structure of the sugar
    • C12N2310/323—Chemical structure of the sugar modified ring structure
    • C12N2310/3233—Morpholino-type ring
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/34—Spatial arrangement of the modifications
    • C12N2310/346—Spatial arrangement of the modifications having a combination of backbone and sugar modifications
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C—CHEMISTRY; METALLURGY
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/351—Conjugate
    • C12N2310/3513—Protein; Peptide
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2310/00—Structure or type of the nucleic acid
    • C12N2310/30—Chemical structure
    • C12N2310/35—Nature of the modification
    • C12N2310/352—Nature of the modification linked to the nucleic acid via a carbon atom
    • C12N2310/3521—Methyl
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2320/00—Applications; Uses
    • C12N2320/30—Special therapeutic applications
    • C12N2320/33—Alteration of splicing

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  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Biomedical Technology (AREA)
  • Chemical & Material Sciences (AREA)
  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Zoology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Physics & Mathematics (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Footwear And Its Accessory, Manufacturing Method And Apparatuses (AREA)
PT167772912T 2015-04-08 2016-04-07 Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão PT3283500T (pt)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562144712P 2015-04-08 2015-04-08

Publications (1)

Publication Number Publication Date
PT3283500T true PT3283500T (pt) 2021-01-28

Family

ID=57073329

Family Applications (1)

Application Number Title Priority Date Filing Date
PT167772912T PT3283500T (pt) 2015-04-08 2016-04-07 Composições e métodos para corrigir distrofia muscular das cinturas tipo 2c com utilização de salto do exão

Country Status (17)

Country Link
US (5) US10273483B2 (zh)
EP (1) EP3283500B1 (zh)
BR (1) BR112017021485A2 (zh)
CA (1) CA2981960C (zh)
CY (1) CY1123599T1 (zh)
DK (1) DK3283500T3 (zh)
ES (1) ES2846902T3 (zh)
HK (1) HK1250720A1 (zh)
HR (1) HRP20210139T1 (zh)
HU (1) HUE052604T2 (zh)
LT (1) LT3283500T (zh)
PL (1) PL3283500T3 (zh)
PT (1) PT3283500T (zh)
RS (1) RS61349B1 (zh)
SI (1) SI3283500T1 (zh)
TN (1) TN2017000427A1 (zh)
WO (1) WO2016164602A1 (zh)

Families Citing this family (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CA2884245C (en) * 2012-09-06 2023-03-14 The University Of Chicago Antisense polynucleotides to induce exon skipping and methods of treating dystrophies
DK3283500T3 (en) * 2015-04-08 2020-11-16 Univ Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping

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DK3283500T3 (en) * 2015-04-08 2020-11-16 Univ Chicago Compositions and methods for correcting limb girdle muscular dystrophy type 2C using exon skipping

Also Published As

Publication number Publication date
HK1250720A1 (zh) 2019-01-11
US20220119820A1 (en) 2022-04-21
EP3283500B1 (en) 2020-11-11
US20180080030A1 (en) 2018-03-22
CA2981960A1 (en) 2016-10-13
US10273483B2 (en) 2019-04-30
LT3283500T (lt) 2020-12-10
BR112017021485A2 (pt) 2018-07-03
ES2846902T3 (es) 2021-07-30
US20230167452A1 (en) 2023-06-01
EP3283500A1 (en) 2018-02-21
CA2981960C (en) 2023-09-19
PL3283500T3 (pl) 2021-05-31
WO2016164602A1 (en) 2016-10-13
US20210032631A1 (en) 2021-02-04
SI3283500T1 (sl) 2020-12-31
CY1123599T1 (el) 2022-03-24
US10801029B2 (en) 2020-10-13
EP3283500A4 (en) 2019-01-23
TN2017000427A1 (en) 2019-04-12
HRP20210139T1 (hr) 2021-03-19
RS61349B1 (sr) 2021-02-26
DK3283500T3 (en) 2020-11-16
HUE052604T2 (hu) 2021-05-28
US20190249180A1 (en) 2019-08-15

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